- Connecticut-based Biohaven on Wednesday said it submitted a treatment for a rare neurodegenerative disease to the Food and Drug Administration for approval.
- The application is for Biohaven’s drug troriluzole, which the company has tested in an inherited condition known as spinocerebellar ataxia that causes problems with movement, speaking and swallowing.
- A year ago, the drug failed to meet its main goal in a Phase 3 study, but subsequent Biohaven analyses of the data turned up more positive signs among participants who had a form of the disease known as SCA Type 3. It’s in this subgroup Biohaven is seeking an approval.
The current version of Biohaven was spun out following the company’s acquisition by Pfizer in a near-$12 billion deal last year. The pharmaceutical company was interested in Biohaven’s migraine treatments, and sent the rest of the biotech’s pipeline to the newly created spinout.
Troriluzole is one of its most advanced candidates, but has previously failed in studies for general anxiety disorder and Alzheimer’s disease. Late-stage testing in obsessive compulsive disorder is ongoing.
In spinocerebellar ataxia, the drug did not significantly improve participants’ scores on a motor function scale by more than those given a placebo in a Phase 3 trial. However, the company reported signs of a treatment effect among those participants who have SCA Type 3, the most common form of spinocerebellar ataxia. Among that group, Biohaven said it observed a “numerical treatment benefit” over placebo.
“The NDA we submitted for troriluzole for SCA3 represents approximately seven years of effort by the Biohaven team to bring forward a potentially new treatment for this ultra-rare disease,” said company CEO Vlad Coric, in a statement. “We look forward to interactions with the U.S. FDA, [European Medicines Agency] and other regulatory agencies across the globe as our submissions advance in the review process."
The company had not previously committed to an FDA submission, noting in an earnings statement earlier this month only that they planned to discuss “next steps” with regulators.
Elsewhere, Biohaven is focusing on a newly licensed TYK2 inhibitor, as well as treatments in epilepsy, bipolar disorder and spinal muscular atrophy.